Cargando…
Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
INTRODUCTION: In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval. OBJECTIVE: The objective of this study was to investigate the opinion of stakeholders about key aspects of rare disease...
Autores principales: | Jonker, Carla J., de Vries, Sieta T., van den Berg, H. Marijke, McGettigan, Patricia, Hoes, Arno W., Mol, Peter G. M. |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8279983/ https://www.ncbi.nlm.nih.gov/pubmed/34091881 http://dx.doi.org/10.1007/s40264-021-01081-z |
Ejemplares similares
-
Registries supporting new drug applications
por: Jonker, Carla J., et al.
Publicado: (2017) -
Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study
por: Jonker, Carla J., et al.
Publicado: (2020) -
Patient Registries: An Underused Resource for Medicines Evaluation: Operational proposals for increasing the use of patient registries in regulatory assessments
por: McGettigan, Patricia, et al.
Publicado: (2019) -
EU-funded initiatives for real world evidence: descriptive analysis of their characteristics and relevance for regulatory decision-making
por: Plueschke, Kelly, et al.
Publicado: (2018) -
Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe
por: Jonker, Carla J., et al.
Publicado: (2022)